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Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1

Respiratory complications are the most common cause of death among patients with Myotonic Dystrophy type 1 (DM1), but the natural history of respiratory decline in DM1 patients is incompletely characterized and few predictors of the progression of respiratory dysfunction have been identified. To ide...

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Autores principales: Hartog, Leigh, Zhao, Jing, Reynolds, Jerry, Brokamp, Gabrielle, Vilson, Ferdinand, Arnold, W. David, LoRusso, Samantha
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8076608/
https://www.ncbi.nlm.nih.gov/pubmed/33927684
http://dx.doi.org/10.3389/fneur.2021.658532
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author Hartog, Leigh
Zhao, Jing
Reynolds, Jerry
Brokamp, Gabrielle
Vilson, Ferdinand
Arnold, W. David
LoRusso, Samantha
author_facet Hartog, Leigh
Zhao, Jing
Reynolds, Jerry
Brokamp, Gabrielle
Vilson, Ferdinand
Arnold, W. David
LoRusso, Samantha
author_sort Hartog, Leigh
collection PubMed
description Respiratory complications are the most common cause of death among patients with Myotonic Dystrophy type 1 (DM1), but the natural history of respiratory decline in DM1 patients is incompletely characterized and few predictors of the progression of respiratory dysfunction have been identified. To identify factors influencing the progression of respiratory dysfunction electronic medical records from 110 adult patients diagnosed with DM1 were reviewed along with data for respiratory symptoms and pulmonary function obtained from routine respiratory therapist clinical evaluations. At baseline, 70.9% had evidence of restrictive respiratory impairment. We examined various parameters of respiratory functional status, and found FVC (% predicted) correlated best with other measures of disease severity. Annual change in FVC was −1.42 (std error = 0.381). Greater CTG repeat size, higher MIRS rating, and longer disease duration were all correlated with lower baseline FVC but not with annual rate of change. Wide variability in clinical phenotype made determination of disease measures directly related to respiratory functional decline challenging.
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spelling pubmed-80766082021-04-28 Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1 Hartog, Leigh Zhao, Jing Reynolds, Jerry Brokamp, Gabrielle Vilson, Ferdinand Arnold, W. David LoRusso, Samantha Front Neurol Neurology Respiratory complications are the most common cause of death among patients with Myotonic Dystrophy type 1 (DM1), but the natural history of respiratory decline in DM1 patients is incompletely characterized and few predictors of the progression of respiratory dysfunction have been identified. To identify factors influencing the progression of respiratory dysfunction electronic medical records from 110 adult patients diagnosed with DM1 were reviewed along with data for respiratory symptoms and pulmonary function obtained from routine respiratory therapist clinical evaluations. At baseline, 70.9% had evidence of restrictive respiratory impairment. We examined various parameters of respiratory functional status, and found FVC (% predicted) correlated best with other measures of disease severity. Annual change in FVC was −1.42 (std error = 0.381). Greater CTG repeat size, higher MIRS rating, and longer disease duration were all correlated with lower baseline FVC but not with annual rate of change. Wide variability in clinical phenotype made determination of disease measures directly related to respiratory functional decline challenging. Frontiers Media S.A. 2021-04-13 /pmc/articles/PMC8076608/ /pubmed/33927684 http://dx.doi.org/10.3389/fneur.2021.658532 Text en Copyright © 2021 Hartog, Zhao, Reynolds, Brokamp, Vilson, Arnold and LoRusso. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Neurology
Hartog, Leigh
Zhao, Jing
Reynolds, Jerry
Brokamp, Gabrielle
Vilson, Ferdinand
Arnold, W. David
LoRusso, Samantha
Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title_full Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title_fullStr Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title_full_unstemmed Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title_short Factors Influencing the Severity and Progression of Respiratory Muscle Dysfunction in Myotonic Dystrophy Type 1
title_sort factors influencing the severity and progression of respiratory muscle dysfunction in myotonic dystrophy type 1
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8076608/
https://www.ncbi.nlm.nih.gov/pubmed/33927684
http://dx.doi.org/10.3389/fneur.2021.658532
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